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CAPN5 genetic inactivation phenotype supports therapeutic inhibition trials
Small molecule pharmacological inhibition of dominant human genetic disease is a feasible treatment that does not rely on the development of individual, patient-specific gene therapy vectors. However, the consequences of protein inhibition as a clinical therapeutic are not well-studied. In advance o...
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| Publicado no: | Hum Mutat |
|---|---|
| Main Authors: | , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7493429/ https://ncbi.nlm.nih.gov/pubmed/31403230 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/humu.23894 |
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